Verastem gets FDA Orphan Drug Designation for Avutometinib and Defactinib

Verastem Oncology announced that the FDA has granted Orphan Drug Designation to avutometinib, a RAF/MEK clamp, in combination with defactinib, a selective FAK inhibitor, for the treatment of pancreatic cancer. At the American Society of Clinical Oncology Annual Meeting in June 2024, Verastem presented initial interim safety and efficacy results from the ongoing RAMP 205 trial of avutometinib and defactinib in combination with current standard of care gemcitabine and nab-paclitaxel in first-line metastatic pancreatic cancer. As of the data cutoff of May 14, 2024, 41 patients had been treated in one of four dose cohort regimens and only patients in dose cohort 1 had a minimum follow up of six months. In the dose level 1 cohort, 83% of patients achieved a confirmed partial response with more than six months of follow up at the time of data cutoff. One dose-limiting toxicity was observed in the dose level 1 cohort, and the dose level was subsequently cleared after additional patients were enrolled. Of the 26 patients in all cohorts who have had the opportunity to have their first scan while on treatment, 21 have experienced a reduction of the change in target lesion sum of diameters.